Collaboration on next-generation AAV gene therapy

30th May 2018

Sirion Biotech and Heidelberg University Hospital sign alliance deal for development of novel AAV capsids and technologies addressing shortcomings of clinical gene therapy trials.

The viral vector specialist Sirion Biotech GmbH today announces the signing of a preferred partnership agreement with Heidelberg University Hospital and AAV pioneer Prof. Dirk Grimm. This strategic alliance will enable both partners to offer engineering of novel AAV capsids for the development of targeted and effective gene therapies, anticipating the growing high demands of clinical gene therapy trials.

In vivo gene delivery was revolutionized by the introduction of AAV-based vectors, which have taken center stage in the development of gene therapies against a wide range of diseases with a genetic component. The tremendous potential of this versatile vector class is undisputed and illustrated by its growing presence in clinical gene therapy trials. While individual applications have already yielded promising results, culminating in the commercialization of two AAV vector-based gene therapies, recent reports from several trials project a growing need for vector optimization technologies to expand the range of AAV target cells and to improve vector and thus patient safety. With a major portion of the vector industry concentrating on questions of large-scale AAV production, there has been only little initiative to unify isolated talent and build a comprehensive, flexible technology platform that is accessible to the drug development industry.

With their new preferred partnership agreement in place, Heidelberg University Hospital’s Prof. Grimm and Sirion Biotech set a milestone for the AAV field, integrating commercial expertise for viral vectors and understanding of the needs of a fast-paced client base, with the newest scientific advances in AAV optimization. The undersigned partnership agreement will help compose consistent long-term projects while maintaining a maximum of flexibility. It enables users to access multiple engineering techniques that help to optimize vector cell and tissue specificities, to boost its productivity and to reduce vector immunogenicity. This will enable customers to obtain and use truly innovative vectors that go beyond the current state-of-the-art and that will be tailored to their specific demands, thereby increasing the customers' chances of success in clinical trials.

“Establishing a fixed cooperation agreement with Prof. Grimm and Heidelberg University Hospital will help us streamline the way we plan and implement our AAV engineering collaborations. Our industrial partners will be able to enter clinical trials months earlier than previously possible, with capsids and vectors optimized for their specified goals,” says Dr. Christian Thirion, CEO and founder of Sirion Biotech “This collaboration with Prof. Grimm offers our customers direct access to the very latest AAV technologies, to maximize the efficiency and safety of their gene therapy programs”, adds Dr. Sabine Ott, VP Business Development & Licensing.
Dr. Dirk Grimm, Professor for Viral Vector Technologies at Heidelberg University Hospital explains: “This preferred partnership agreement underpins our long-lasting fruitful collaboration with SIRION and accelerates our efforts to leverage our expertise in AAV capsid optimization toward the clinic for a variety of disorders“.

Sirion Biotech specializes in custom engineering services for viral vectors in the life sciences and industry.

Prof. Grimm is an expert in AAV vector design and engineering who pioneered the use of molecular evolution technologies for generation of AAV capsids with altered cell specificities and other improved properties.





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